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The medullary, arm, leg, and/or respiratory function of patients with amyotrophic lateral sclerosis (ALS) is affected in several ways
.
The Revised ALS Functional Rating Scale (ALSFRS-R) is the most commonly used method for assessing these areas
For example, not all patients with symptomatic medulla oblongata develop leg symptoms, even in the final stages of the disease
.
In fact, most patients who die from ALS have an ALSFRS-R total score above 0, suggesting that some symptoms may never appear during the disease process
place
An online questionnaire was constructed using a cloud-based clinical data management platform (Castor EDC, V.
2021.
2, https://
.
The questionnaire consisted of a validated self-report version of ALSFRS-R.
An online questionnaire was constructed using a cloud-based clinical data management platform (Castor EDC, V.
Schematic diagram of the probability of winning by patient intent
Schematic diagram of the probability of winning by patient intentUltimately, all treated patients were compared to all placebo patients
.
This produces a matrix with rows equal to the number of patients treated and columns equal to the number of placebo patients
Ultimately, all treated patients were compared to all placebo patients
Patient preferences varied widely among the 433 participants
In conclusion, the PROOF endpoint provides a balanced patient-centered analysis of functional improvement that may help refine the risk-benefit assessment of new ALS therapies
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